Li Ou, PhD

Assistant Professor, Department of Pediatrics

Li Ou

Contact Info

Office Phone 612-625-6912

Fax 612-624-2682

Office Address:
Pediatric Genetics and Metabolism
13-122 PWB
516 Delaware St. SE
Minneapolis MN 55455

Mailing Address:
Pediatric Genetics and Metabolism
516 Delaware St. SE MMC 391
Minneapolis MN 55455

Lab Address:
420 Washington Ave SE
5-178 MCB
Minneapolis MN 55455

PhD, Genetics, University of Minnesota, Minneapolis, MN

BS, Biotechnology, Sichuan University, Chengdu, Sichuan, China


Dr. Li Ou is currently an Assistant Professor in the Department of Pediatrics, University of Minnesota. He received his PhD (major in Genetics, minor in Biostatistics) at the University of Minnesota. Dr. Ou’s previous work on zinc finger nuclease mediated gene editing for treating MPS I and MPS II diseases led to two Phase I/II clinical trials (NCT02702115 and NCT03041324). These are the first two clinical trial in the world approved for the application of in vivo gene editing to treat genetic disorders. Dr. Ou joined the Department of Pediatrics Faculty as an Assistant Professor in 2019. 

Awards & Recognition

  • Innovator in Translational Research Award, Department of Pediatrics, University of Minnesota (2020)
  • Pediatric Research, Education and Scholarship Symposium Presentation Award, Department of Pediatrics, University of Minnesota (2013, 2018)
  • WORLD Symposium Young Investigator Award (2016, 2017)
  • Conference on Clinical Research for Rare Diseases Travel Award (2016)
  • American Society of Gene and Cell Therapy Meritorious Abstract Travel Award (2016)
  • Doctoral Dissertation Fellowship, University of Minnesota (2015)
  • UCLA Cross-disciplinary Scholars in Science and Technology Fellowship (2010)
  • Baosteel Fellowship for Excellent Students (2009)
  • Sichuan University Freshman Fellowship (2007)


Research Summary/Interests

  • Gene therapy, gene editing
  • Rare diseases


  1. Ou L*, Przybilla MJ, Ozan A, Kim S, Overn P, Jarnes J, O’Sullivan MG, Whitley CB. A highly efficacious gene editing system corrects metabolic and neurological complications of Hurler syndrome. Mol Ther. 2020. (*corresponding author)
  2. Ou L*, Przybilla MJ, T?b?ran A, Overn P, O’Sullivan MG, Jiang X, Sidhu R, Kell PJ, Ory DS, Whitley CB. A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases. Gene Ther. 2020 Jan 2. doi: 10.1038/s41434-019-0120-5. (*corresponding author)
  3. Ou L*, Kim S, Whitley CB, Jarnes-Utz JR. Genotype-phenotype correlation of gangliosidosis mutations using in silico tools and homology modeling. Mol Genet Metab Rep. 2019 Jul 17;20:100495. (*corresponding author)
  4. Ou L, DeKelver R, Rhode M, Tom S, Radeke R, St Martin SJ, Santiago Y, Sproul S, Przybilla MJ, Koniar BL, Podetz-Pedersen KM, Laoharawee K, Cooksley RD, Meyer KE, Holmes MC, McIvor RS, Wechsler T, Whitley CB. ZFN-mediated in vivo genome editing corrects murine Hurler syndrome. Mol Ther. 2019 Jan 2;27(1):178-187.
  5. Ahmed A, Ou L, Rudser K, Eisengart J, Whitely CB. A longitudinal study of neurocognition and behavior in patients with Hurler-Scheie syndrome heterozygous for the L238Q mutation. Mol Genet Metab Rep. 2019 Jun 27;20:100484.
  6. Przybilla MJ, Ou L, T?b?ran A, Jiang X, Sidhu R, Kell PJ, Ory DS, O'Sullivan MG, Whitley CB. Comprehensive behavioral and biochemical outcomes of novel murine models of GM1-gangliosidosis and Morquio syndrome type B. Mol Genet Metab. 2019 Feb;126(2):139-150.
  7. Ou L*, Przybilla MJ, Whitley CB. Metabolomics profiling reveals profound metabolic impairments in mice and patients with Sandhoff disease. Mol Genet Metab. 2019 Feb;126(2):151-156. (*corresponding author)
  8. Laoharawee K, DeKelver R, Podetz-Petersen KM, Rohde M, Sproul S, Nguyen HO, Nguyen T, St Martin S, Ou L, Tom S, Radeke R, Meyer KE, Holmes MC, Whitley CB, Wechsler T, McIvor RS. Dose-dependent IDS expression and prevention of metabolic and neurologic disease in a mouse model of Hunter Syndrome by ZFN-mediated in vivo genome editing. Mol Ther. 2018. 26 (4), 1127-1136.
  9. Ou L, Przybilla MJ, Koniar BL, Whitley CB. RTB lectin-mediated delivery of lysosomal ?-L-iduronidase mitigates disease manifestations systemically including the central nervous system. Mol Genet Metab. 2018 Feb;123(2):105-111.
  10. Ou L*, Przybilla MJ, Koniar BL, Whitley CB. SAAMP 2.0: an algorithm to predict genotype-phenotype correlation of lysosomal storage diseases. Clin Genet. 2018 May;93(5):1008-1014. (*corresponding author)
  11. Schadewald A, Kimball E, Ou L. Coping strategies, stress, and support needs in caregivers of children with mucopolysaccharidosis. JIMD Reports. 2018;42:89-97. 
  12. Ou L*, Przybilla MJ, Whitley CB. Phenotype prediction for mucopolysaccharidosis type I by in silico analysis. Orphan J Rare Dis. 2017 Jul 4;12(1):125. (*corresponding author)
  13. Ou L*, Przybilla MJ, Whitley CB. Proteomic analysis of mucopolysaccharidosis I mouse brain with two-dimensional polyacrylamide gel electrophoresis. Mol Genet Metab. 2017 Jan-Feb;120(1-2):101-110. (*corresponding author)
  14. Ou L, Przybilla MJ, Koniar BL, Whitley CB. Elements of lentiviral vector design study toward gene therapy for treating mucopolysaccharidosis I. Mol Genet Metab Rep. 2016 Sept; 8:93-97.
  15. Ou L, Herzog T, Koniar BL, Gunther R, Whitley CB. High-dose enzyme replacement therapy in murine Hurler syndrome. Mol Genet Metab. 2014 Feb;111(2):116-22.
  16. Ou L, Herzog TL, Wilmot CM, Whitley CB. Standardization of ?-L-iduronidase enzyme assay with Michaelis-Menten kinetics. Mol Genet Metab. 2014 Feb;111(2):113-5.
  17. Ou L, Duan D, Wu J, Nice E, Huang C. The application of high throughput siRNA screening technology to study host-pathogen interactions. Comb Chem High Throughput Screen. 2012 May 1;15(4):299-305.
  18. Ou L, Wang X, Zou F. Is iPS cell the panacea? IUBMB Life. 2010 Mar;62(3):170-5.